Stem Cell Therapies for Epidermolysis Bullosa Treatment

  • Epidermolysis bullosa (EB) is a rare skin disease characterized by skin fragility and bullous formation. 
  • Treatment focuses on improving symptoms with topical treatments to avoid complications and infections. 
  • Stem cells, undifferentiated cells capable of producing, maintaining, and replacing terminally differentiated cells and tissues, have been used for the treatment of severe forms of the disease. 
  • Stem cells from different sources, mainly hematopoietic and mesenchymal, autologous or heterologous, have been used for the treatment of the most severe forms of the disease. 
  • The mechanisms through which stem cells exert their beneficial role are still unknown or incompletely understood. 
  • The transplantation of skin grafts to patients produced by gene-corrected autologous epidermal stem cells has been successful for the treatment of skin lesions in a limited number of patients. 
  • However, these treatments do not address the internal epithelia-related complications manifested in patients with more severe forms.

 


Epidermolysis Bullosa (EB) Overview 

 

  • EB is a genodermatopathies characterized by blister formation due to skin fragility and other epithelial tissues. 
  • Clinical manifestations range from localized to generalized blisters and lesions. 
  • Secondary deficits include chronic wounds, scars, deformities, infections, and complications to multiple internal organs. 
  • The skin is the main organ affected, but other epithelial tissues are often implicated. 
  • EB is a multifactorial and multisystemic disease with significant mortality and morbidity rates. 
  • Subtypes result from mutations in genes encoding various proteins crucial for maintaining structural stability and strong adhesion of keratinocytes. 
  • Diagnosis and categorization are based on personal and family history, immunomapping, transmission electron microscopy, and molecular techniques. 
  • Recent research has initiated clinical trials of new cell- and gene-based therapies. 
  • EB was first described in 1886 and is classified into four main types: simplex (SEB), junctional (JEB), dystrophic (DEB), and mixed.
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